WOBURN, Mass., April 30, 2025 -- Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced today that preclinical data will be presented on its proprietary RSwitch technology, which enables the fine-tuning of transgene levels in gene and cell therapy applications, at the American Society of Gene And Cell Therapy (ASGCT) 28th Annual Meeting, which will be held from May 13 -17th, 2025, in New Orleans, LA.
Title: RSwitch Enabled Gene Therapy to Fine Tune Frataxin Expression for the Treatment of Friedrich's Ataxia
Authors: Samuel A. Hasson, Jon Dempersmier, Mariam Elhawary, Diane Hamann, Ian McLachlan, Chris Yates, Travis Wager, and Simon Xi
Session date and time: Wednesday, May 14, 2025, 5:30 - 7:00 PM CT
Location: Poster Hall I2
Poster ID: AMA251
About RSwitch
RSwitch is a proprietary regulatable gene therapy system that enables oral, small molecule drug control of transgene levels in gene and cell therapy applications. RSwitch encodes a "dimmer" switch that makes the expression of transgene dependent on the administration of an oral small molecule drug that controls the system. Only when the drug is administered is the system activated. Furthermore, the level of gene expression is dependent on how much drug is administered. This precise gene control has the potential to enable fine control of the expression of a therapeutic protein. Rgenta has demonstrated the RSwitch system's feasibility in vitro and in vivo, achieving dose-dependent expression of reporter transgenes following small molecule administration. RSwitch technology offers versatile control across multiple gene and cell therapy applications, and the company is actively exploring strategic partnerships.
About Rgenta Therapeutics
Rgenta Therapeutics is a clinical stage biotechnology company developing a pipeline of oral RNA-targeting small molecule medicines with an initial focus on oncology and neurological disorders. Our proprietary platform mines the massive genomics data to identify targetable RNA processing events and design small-molecule glues to modulate the interactions among the spliceosome, regulatory proteins, and RNAs. Our lead programs and unique approach are unlocking the therapeutic potential of historically undruggable targets in human diseases. Learn more at: http://www.rgentatx.com.
Contacts
Investors:
Sylvia Wheeler
Wheelhouse Life Science Advisors
Swheeler@wheelhouselsa.com
Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
Lwolffe@wheelhouselsa.com
Media
Aljanae Reynolds
Wheelhouse Life Science Advisors
Areynolds@wheelhouselsa.com
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